NCT02124174

Vidaza and Valproic Acid Post Allogeneic Transplant for High Risk AML and MDS

Maintenance Therapy With Azacitidine and Valproic Acid After Allogeneic Stem Cell Transplant in Patients With High-Risk Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS)(Version 1_06 Jan 2012)

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Patrick Stiff · Academic / Other
• Sex: All
• Age: 2 Years – 89 Years
• Healthy volunteers: Not accepted

Summary

Phase II trial combining azacitidine with valproic acid as maintenance therapy post allogeneic stem cell transplantation in patients with high-risk MDS/AML. We hypothesize that adding valproic acid to azacitidine will improve outcomes via both direct anti-tumor and immunologically mediated antitumor response with alloreactive donor lymphocytes, having an additive effect and extending 1 year survival in patient with high-risk AML/MDS after hematopoietic stem cell transplant. Based on aforementioned data from the US Department of Health and Human Services, standard 1 year survival for AML after stem cell transplant is near 40%. We hypothesize that valproic acid and azacitidine will prolong survival, with a 1 year survival goal of 60%. In addition to assessing for 1 year survival, we will have secondary objectives of assessing progression-free survival, relapse, and toxicity. The primary toxicity endpoint from this will be cytopenias and infections.

Detailed description

To assess the combination of valproic acid and azacitidine in preventing relapse in patients with high-risk Acute Myeloid Leukemia (AML) and myelodysplastic syndrome (MDS) after allogeneic stem cell transplant. The primary objective of this study will be determining the 1 year overall survival from combining valproic acid (VPA) with 5-azacytidine (5-aza). To assess the effect that adding valproic acid to azacitidine will have in patient with high-risk Acute Myeloid Leukemia (AML) and myelodysplastic syndrome (MDS) after allogeneic stem cell transplant on the following endpoints

Conditions

• Acute Myelogenous Leukemia AML
• Myelodysplastic Syndrome MDS

Interventions

Type	Name	Description
DRUG	Vidaza and Valproic Acid	Days 1-5: 5-Azacytidine 40 mg/m\^2 daily Days 1-5: +Valproic acid 15 mg/kg daily Days 6-28: Valproic acid 15 mg/kg daily \*treatments will be repeated on the same days of each cycle for up to 4 total cycles. Each cycle will consist of 28 days.

Timeline

• Start date: 2012-01-01
• Primary completion: 2022-01-01
• Completion: 2022-01-01
• First posted: 2014-04-28
• Last updated: 2021-04-26

Locations

1 site across 1 country: United States